New compound prevents onset of type 1 diabetes in animal models

A team of scientists has tested a potent synthetic compound that prevents type 1 diabetes in animal models of the disease.

Lead author Laura Solt from the Florida campus of The Scripps Research Institute (TSRI) said that the animals in the study never developed high blood sugar indicative of diabetes and beta cell damage was significantly reduced compared to animals that hadn't been treated with the compound.

Type 1 diabetes is a consequence of the autoimmune destruction of insulin-producing beta cells in the pancreas. While standard treatment for the disease aims to replace lost insulin, the new study focuses instead on the possibility of preventing the initial devastation caused by the immune system, stopping the disease before it even gets started.

In the study, the scientists tested an experimental compound known as SR1001 in non-obese diabetic animal models. The compound targets a pair of "nuclear receptors" (ROR alpha and ROR gamma) that play critical roles in development of a specific population (Th17) of immune cells associated with the disease.

Solt added that because Th17 cells have been linked to a number of autoimmune diseases, including multiple sclerosis, they thought their compound might inhibit Th17 cells in type 1 diabetes and possibly interfere with disease progression and they were right.

The researchers found SR1001 eliminated the incidence of diabetes and minimized insulitis, which is the inflammation associated with, and destroyer of, insulin-producing cells, in the treated animals. The compound suppressed the immune response, including the production of Th17 cells, while maintaining normal insulin levels; it also increased the frequency of the expression of Foxp3 in T cells, which controls the development and function of a type of immune cell known as T regulatory cells.

Solt notes that the study strongly suggests that Th17 cells have a pathological role in the development of type 1 diabetes and use of ROR-specific synthetic compounds targeting this cell type may have potential as a preventative therapy for type 1 diabetes. It certainly opens the door for other areas to be looked at.

The study appears in the journal Endocrinology.